Long-term results of phase II trial of apatinib for progressive radioiodine refractory differentiated thyroid cancer.

Radioiodine refractory differentiated thyroid cancer (RAIR-DTC) has been a global challenge due to its poor prognosis and limited treatment options.To report the long-term results of the phase II clinical trial of apatinib, an anti-angiogenic tyrosine kinase inhibitor, for RAIR-DTC.Open-label, exploratory phase II clinical trial among progressive RAIR-DTC patients.Apatinib treatment once daily until disease progression, unmanageable toxicity, withdrawal, or death.The primary end points were objective response rate (ORR) and disease control rate (DCR). Progression-free survival (PFS), overall survival (OS), duration of response, long-term safety and the association between patients with different tumor genotype (BRAF  V600E and TERT promotor mutation) and their PFS were also assessed.The ORR was 80%, and the DCR was 95%. The overall median PFS was 18.4 months (95% confidence interval [CI], 9.2-36.8 months) and median OS was 51.6 months (95%CI, 29.2-not reached [NR]). Patients with BRAF  V600E mutation (10 of 18 evaluated) had a longer median PFS compared with patients with BRAF wild-type (NR vs. 9.2 months, P=0.002). The most common adverse events included palmar-plantar erythrodysaesthesia syndrome (19/20), proteinuria (18/20) and hypertension (16/20).In this long-term evaluation, apatinib displayed sustainable efficacy and tolerable safety profile, warranting it as a promising treatment option for progressive RAIR-DTC.

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Authors: Yan-Song Lin, Xin Zhang, Chen Wang, Yan-Qing Liu, Wen-Min Guan, Jun Liang